Hoth Therapeutics Issues Comprehensive Pipeline Update Highlighting Clinical Progress, Orphan-Designated HT-KIT, Advancing HT-001 Phase 2, and New GDNF Metabolic Program
Rhea-AI Summary
Hoth Therapeutics (NASDAQ: HOTH) issued a pipeline update highlighting clinical and preclinical progress across four programs: HT-001 (topical EGFR-inhibitor rash therapy) is in Phase 2 CLEER-001 with a reported strong safety profile and consistent improvement trends; HT-KIT received FDA Orphan Drug Designation and is completing IND-enabling toxicology with an IND targeted in 2026; HT-ALZ is progressing GLP, PK, and CNS-penetration work toward a regulatory package in 2026; and a newly launched GDNF-based metabolic program (collaboration with Atlanta VA) has begun preclinical work with early data expected in 2026.
The company also reports manufacturing scale-up, expanded IP filings, and ongoing academic and AI-assisted collaborations, with near-term milestones including CLEER-001 data updates and HT-KIT IND completion.
Positive
- HT-001 Phase 2: no dose-limiting toxicities reported
- HT-KIT: FDA Orphan Drug Designation secured
- HT-KIT: IND-enabling toxicology moving toward completion
- GDNF program: VA collaboration initiated with study activities underway
- HT-ALZ: GLP and PK studies producing supportive CNS-penetration data
Negative
- No completed Phase 2 efficacy readout reported yet for HT-001
- IND for HT-KIT not filed; IND submission expected in 2026
- Early-stage GDNF program: preclinical data only, with first results expected in 2026
Insights
Pipeline moves (Phase 2 progress, orphan designation, IND prep and new VA collaboration) materially advance clinical and regulatory timelines.
Hoth shows measurable clinical progress: a Phase 2 program for HT-001 reporting no dose‑limiting toxicities and consistent symptom improvements strengthens its safety/efficacy signal in supportive oncology care, while HT-KIT already holds Orphan Drug Designation and is completing IND‑enabling studies toward an
Key dependencies and risks include completion of formal toxicology and manufacturing data for the IND, maturation of GLP/PK/BBB packages for HT-ALZ, and reproducibility of early efficacy signals; the new GDNF VA collaboration accelerates preclinical throughput but relies on institutional timelines. Watch for the CLEER‑001 Phase 2 data update, IND filing for HT-KIT and early GDNF results expected in
Pipeline Highlights
HT-001 (Topical Epidermal Growth Factor Inhibitor) – Phase 2 CLEER-001 Trial Advancing
Hoth continues to advance HT-001 in its Phase 2 CLEER-001 clinical trial targeting EGFR-inhibitor–associated rash, a significant unmet need in oncology supportive care.
Recent progress includes:
- Strong safety profile to date with no dose-limiting toxicities observed.
- Consistent improvement trends in rash severity and pruritus
- Increased clinical-site engagement and favorable investigator feedback.
A further clinical update is anticipated in the coming months as enrollment continues to progress.
HT-KIT (Orphan Drug Designation for Mast Cell Diseases) – IND Preparation Underway
HT-KIT, Hoth's targeted KIT-inhibitor program for mastocytosis and related mast-cell–driven diseases, continues to progress through IND-enabling activities.
Key accomplishments:
- FDA Orphan Drug Designation already granted.
- Compelling preclinical efficacy showing potent KIT inhibition and suppressed mast-cell activation.
- IND-enabling toxicology studies moving toward completion.
- Ongoing manufacturing scale-up and analytical characterization
Hoth expects to finalize its' IND submission in 2026, followed by first-in-human studies.
HT-ALZ (Therapeutic for Alzheimer's Disease) – Advancing Through GLP and PK Development.
HT-ALZ continues to deliver supportive data across absorption, distribution, and neuroinflammatory pathways.
Recent highlights:
- GLP studies underway with positive PK, biodistribution, and CNS-penetration modeling
- Regulatory-facing package expected to mature in 2026.
GDNF-Based Weight-Loss & Metabolic Program (VA Collaboration) – Newly Accelerated Initiative
Hoth's newest program leverages GDNF (Glial-Derived Neurotrophic Factor) to target obesity, hepatic steatosis, and metabolic dysfunction, representing one of the largest and fastest-growing therapeutic markets globally.
Recent achievements:
- Study preparations initiated with the Atlanta VA Medical Center
- Mouse procurement completed; high-fat-diet regimen launches per approved VA protocols.
- Aim 1 of the research program now underway, with early data expected in 2026.
- Program supported by strong academic collaboration and growing IP protection.
Expanding Intellectual Property & Strategic Collaborations
Hoth continues to broaden its IP position through new filings and expanded protection around:
- HT-001 dermatology and oncology-supportive-care mechanisms
- HT-KIT mast-cell-disease formulations, methods, and manufacturing
- HT-ALZ CNS-focused data and delivery systems
- GDNF weight-loss and hepatic-function applications
The Company maintains collaborations with leading institutions, including the Atlanta VA Medical Center, academic neuroscience groups, and AI-assisted discovery platforms.
Upcoming Milestones
- CLEER-001 Phase 2 HT-001 clinical data update
- Completion of toxicology and IND filing for HT-KIT
- GLP, BBB, and PK updates for HT-ALZ
- VA metabolic program early findings
Management Commentary
Robb Knie, Chief Executive Officer of Hoth Therapeutics, commented:
"Our therapeutic pipeline has never been more focused or better positioned. With HT-001 advancing in the clinic, HT-KIT nearing IND submission, HT-ALZ progressing through GLP development, and our new GDNF program targeting one of the largest markets in medicine."
About Hoth Therapeutics, Inc.
Hoth Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, impactful, and ground-breaking treatments with a goal to improve patient quality of life. We are a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing. Utilizing a patient-centric approach, we collaborate and partner with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options. To learn more, please visit https://ir.hoththerapeutics.com/.
Forward-Looking Statement
This press release includes forward-looking statements based upon Hoth's current expectations, which may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws, and are subject to substantial risks, uncertainties, and assumptions. These statements concern Hoth's business strategies; the timing of regulatory submissions; the ability to obtain and maintain regulatory approval of existing product candidates and any other product candidates we may develop, and the labeling under any approval we may obtain; the timing and costs of clinical trials, and the timing and costs of other expenses; market acceptance of our products; the ultimate impact of the current coronavirus pandemic, or any other health epidemic, on our business, our clinical trials, our research programs, healthcare systems, or the global economy as a whole; our intellectual property; our reliance on third-party organizations; our competitive position; our industry environment; our anticipated financial and operating results, including anticipated sources of revenues; our assumptions regarding the size of the available market, benefits of our products, product pricing, and timing of product launches; management's expectation with respect to future acquisitions; statements regarding our goals, intentions, plans, and expectations, including the introduction of new products and markets; and our cash needs and financing plans. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. You should not place reliance on these forward-looking statements, which include words such as "could," "believe," "anticipate," "intend," "estimate," "expect," "may," "continue," "predict," "potential," "project" or similar terms, variations of such terms, or the negative of those terms. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, the Company cannot guarantee such outcomes. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described in the section titled "Risk Factors" in Hoth's most recent Annual Report on Form 10-K and Hoth's other filings made with the U. S. Securities and Exchange Commission. All such statements speak only as of the date made. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates, and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance, or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events, or circumstances or to reflect the occurrences of unanticipated events, except as may be required by applicable law.
Investor Contact:
LR Advisors LLC
Email: investorrelations@hoththerapeutics.com
www.hoththerapeutics.com
Phone: (678) 570-6791
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SOURCE Hoth Therapeutics, Inc.
FAQ
What progress did Hoth Therapeutics (HOTH) report for HT-001 in the Phase 2 CLEER-001 trial?
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